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Treatment of a retinal dystrophy, fundus albipunctatus, with oral 9-cis-β-carotene
  1. Ygal Rotenstreich1,4,
  2. Dror Harats2,4,
  3. Aviv Shaish2,
  4. Eran Pras3,4,
  5. Michael Belkin1,4
  1. 1Maurice and Gabriela Goldschleger Eye Research Institute, Sheba Medical Center, Tel-Hashomer, Israel
  2. 2The Bert W Strassburger Lipid Center, Sheba Medical Center, Tel-Hashomer, Israel
  3. 3Department of Ophthalmology, Assaf Harofeh Medical Center, Zerifin, Israel
  4. 4Sackler School of Medicine, Tel-Aviv University, Tel-Aviv, Israel
  1. Correspondence to Dr Ygal Rotenstreich, Maurice and Gabriela Goldschleger Eye Research Institute, Sheba Medical Center, Tel-Hashomer 52621, Israel; Ygal.rotenstreich{at}


Background Fundus albipunctatus is a retinal dystrophy caused by a mutation in the gene encoding 11-cis-retinol dehydrogenase which delays the recovery of rod photoreceptor cells from light stimulation leading to night blindness. A recent study of a mouse model of fundus albipunctatus treated with 9-cis-retinal showed an improvement in visual function and structure.

Methods Seven patients with fundus albipunctatus were given a daily food supplement of four capsules containing high-dose 9-cis-β-carotene for 90 days. The subjects were tested before and after treatment by visual field and electroretinogram in both eyes. This non-randomised prospective phase I study was registered at (NCT00478530).

Results All patients showed significant improvements in peripheral visual field (mean deviation improved from −4.77±2.0 to −3.28±2.28, p=0.009, t test) and a highly significant improvement in rod recovery rates measured electroretinographically (maximal scotopic b-wave amplitude responses, improved from 197±49 μV to 292±48 μV, p<0.001, t test). No complications or side effects were observed.

Conclusion Oral treatment with 9-cis-β-carotene led to reversal of a human retinal dystrophy. This potential therapy is readily available and should be evaluated in retinal dystrophies of similar mechanisms such as various types of retinitis pigmentosa.

  • Retina
  • vision
  • electrophysiology
  • treatment medical
  • dystrophy

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  • DH & MB contributed equally to this work.

  • Funding Maratier Fund.

  • Competing interests Tel-Hashomer Medical Center applied for a patent for the ophthalmic uses of the compound tested.

  • Patient consent Obtained.

  • Ethics approval Ethics approval was provided by the Sheba Medical Center Institutional Review (Helsinki) Committee.

  • Provenance and peer review Not commissioned; externally peer reviewed.

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